The Food and Drug Administration has issued a guidance document detailing a possible new regulation against off-label use of a category of medical devices, a development that stunned the device industry.

The new FDA regulation would extend the legal doctrine called "intended use" to cover manufacturers of medical devices and make those manufacturers legally liable for off-label uses of their products.

The FDA’s decision could have profound and deadly consequences for individuals' health and decimate the medical device industry, says Paul Citron, a founding member of the American Institute for Medical and Biological Engineering. Under the proposed new regulation, if a doctor used a medical device in a way that the label did not suggest, the manufacturer of the device could be held liable.

Clampdown on Physicians

Citron says he is aghast at what the FDA is attempting to do.

Innovation in device use “would become legally much more difficult to do if the FDA succeeds in their new regulation of medical device manufacturers,” Citron said. “In fact, many medical devices that Americans benefit from in helping them cope with a specific ailment were found to help with those ailments through off-label use—in part because physicians are a self-regulating, communicating body.”

Citron cites the clinical defibrillator as a case in which a medical device was used in an off-label way that then became the on-label way.

“Indications for use of the defibrillator slowly became broader and broader and broader, while the FDA report had it that it is for a particular therapy use. Through off-label use, with more patients using it, physicians in that scenario were using the device in an off-label way, but then it became the label view,” Citron said. “It had an approved label, but then physicians would do studies on their own, and as word got out that this has been proven to be useful for this broader group of patients, even though we don't have an official indicator, physicians started using it off-label, even though companies couldn't promote such use.”

Stops Doctors’ Creativity, Innovation

The proposed FDA guideline would specifically target "in vitro diagnostic" (IVD) medical devices which are categorized as "for research use only" (RUO) or "for investigational use only" (IUO). Paul Howard, a senior fellow at the Manhattan Institute, says part of the motivation for the FDA's move might be an inherent discomfort with the leeway doctors have in prescribing treatment.

“Off-label use is critically important. Once the drug or device is out there, physicians will use it in ways the agency and manufacturers never even intended. These are highly trained medical physicians using their best clinical judgment, and for areas where there are not many treatments available,” Howard explains.

“Yes, ‘off-label’ sounds ‘back of the kitchen sink’, but these are extremely well-trained medical professionals—they look at 'this hits this chemical target, this affects the immune system.'. They validate in the literature; they say, 'My patient might benefit; let me try that.' In the cancer and autoimmune space, this is particularly important. Half or more [of all] cancer therapies are off-label—lupus has only one therapy approved. Off-label is a critical safety valve,” Howard says.

Expects Industry Omniscience

Citron notes the proposed regulation would punish industry for use of their devices in a way they cannot easily monitor or affect.

“Current regulations say that manufacturers are not allowed to promote the off-label use of a device. Nothing keeps the physicians from off-label use, but this [proposed regulation] would penalize industry for their innovations,” Citron states.

“The twist on it is that it is for investigational devices,” he added. “Imagine a physician who uses the investigational device in his hands that is supposed to be used for Investigational Use A, but the physician has a patient with Syndrome B, and they think it might be appropriate to try. The company is not endorsing that, or suggesting it, and may not even be aware that physician is applying it. The FDA apparently expects the company to be all-seeing, ever-present, if they plan to hold them liable for such action.”

Faulted As Overregulation

Howard says one reason off-label use has become a factor in the advance of medical technology in the United States is because the FDA has an approval process that does not make any sense.

“The FDA’s process for ‘new label indicators’ is extremely time-consuming and expensive, so many of the number of available therapies and patients we now have come off-label. It is part standard medical practice,” Howard said. “In other areas, the FDA recognizes off-label use is valuable. The physician community would revolt tremendously if they sought to end off-label use across the board.”

Citron points out physicians already work around the FDA's cumbersome and antiquated procedures in attempts to benefit patients.

“Often the peer-review process will accept papers containing data for use of a therapy that is still off-label, and it may not happen for another year or three for it to become on-label, but the physicians announce to each other for a broader application of a device,” Citron said. “The clinical community is excellent at communicating with each other to improve care.”